On February 13th, Marathon Pharmaceuticals announced that it will hold off on the launch of its drug Emflaza, which was approved by the FDA on February 9th. [1,2,3] The drug, a corticosteroid known generically as deflazacort, has been available across Europe and in Canada for decades, and is used to treat Duchenne muscular dystrophy. [2,4] The rare genetic disease, which affects roughly 15,000 patients of whom most are male, causes the gradual destruction of the muscles. [2,4] Patients first lose their ability to walk, and most die in their 20s or 30s due to heart muscle failure. [2,5] While Emflaza and its generic version deflazacort do not cure the disease, the drugs do appear to improve muscle strength, according to the FDA in a statement published following the approval of Emflaza. [6]

After the approval of Emflaza, Marathon Pharmaceuticals, an Illinois-based company, planned to list the drug for $89,000 dollars a year, to the dismay of many patients, lawmakers, and advocacy groups. [2,4] Eric Messner, vice president of sales and marketing for Marathon, commented that the drug would cost between $50,000 to $54,000 following all rebates and discounts. [3] However, patients have been importing deflazacort for years from other countries for a mere $1,200 a year, meaning that even following price reductions, Marathon’s drug is a 4,400 percent markup. [2,3]

While rare diseases were once viewed as unprofitable, government incentives beginning in 1983 changed the landscape for drug sales by providing major benefits to companies selling treatments for “orphan” diseases affecting fewer than 200,000 Americans. [2] These changes mean that presently, orphan drugs can bring about a considerable financial gain for manufacturers like Marathon. Due to the government incentives, Marathon will now be the sole US distributor of deflazacort for seven years to encourage research for new treatments for the disease, leading to concerns from government officials as well as private citizens. [3,5]

Earlier this week, Sen. Bernie Sanders and Rep. Elijah Cummings sent a letter to the company in hopes of interrogating the expensive price tag of the drug. [1] “We believe Marathon is abusing our nation’s 'orphan drug' program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases,” the politicians stated. [1] “Marathon will have a monopoly on deflazacort for years to come, preventing less expensive generic competitors from entering the market, despite the fact that this drug is already available in generic form in other countries.” [2] The politicians also expressed their concern that the drug, which can be used to treat other diseases, will be used for condition it isn't approved for in order to boost sales. [2] Sanders later took to Twitter to comment on the “outrageous” price posed by Marathon. [1]

Following these pricing concerns, Marathon CEO Jeffrey Aronin announced in a statement published by the advocacy group CureDuchenne that the company would delay the release of its product and meet with caregivers to explain commercialization plans and address concerns about the process. [1,2] “We are pausing our launch, which has not yet taken place. We have not sold any new product and will pause that process,” said Aronin. [3] However, Aronin defended the company’s plans, stating, “Our goal in commercializing Emflaza all along has been to make it available to that broader set of patients who, prior to FDA approval, have not had access to the therapy.” [3]

While the conditions of commercialization are under review, patients may still get the drug via the company’s access program, and those who have been obtaining the drug from other countries will be able to continue to do so. [2,3 ]However, once the drug is available in the US, the only way for Americans to legally purchase the drug will be to buy it from Marathon, according to the FDA. [5] Still, many patients worry about the extremely high cost of the drug, although the Marathon promises very low out-of-pocket-costs due to their insurance and financial assistance offerings. [2] "We expect patients will pay a standard co-pay of typically $20 or less per prescription," says Aronin, who is confident that every patient who needs the drug will be able to access it affordably. [5] However, individuals like Joanne Wechsler, a parent advocate with Parent Project Muscular Dystrophy, worry that their insurance may not cover the medication: "My big concern is ... I have to fight with my insurance company over everything." [5]

While many still question the reasoning behind the $89,000 pricetag, Marathon has justified its decision to list the drug at such a high price. "There are some people who think you can just bring a generic from Europe and sell it in the United States, and that's simply not the case," said Wanda Moebius, Marathon’s Vice President of Communications. [5] Furthermore, Aronin commented that the company needs to gain back the money invested in clinical studies required to bring the drug to the US.5 Yet, Sanders and Cummings, in their letter, noted that Marathon relied upon previous data dating back to the 1990s that proved the drug worked, leading to speculation about the legitimacy of Aronin’s defense. [5,6] According to Aronin, the price of the drug would also augment Marathon’s “ability to fund future research and development,” but researchers at Harvard University, in August of last year, found no evidence of a relationship between how much large pharmaceutical companies charge for drugs and how much they invest into research. [5] The study did not include Marathon, but the frequent justification of “research and development” used by drug companies is seen by some as a way to evade the question of drug pricing. [5]

Still, the accessibility and benefits of Emflaza are promising. Patients walk an average of three more years with the drug, can breathe without an aid for longer, and develop scoliosis at a lower rate as a result of stronger back muscles. [5] Additionally, the drug is more effective and comes with milder side effects compared to prednisone, the next-best treatment which is regularly available in the US. [4,5] Amidst these plentiful benefits, Aronin retains that Marathon is altruistic in its mission: "Please know we sought FDA approval of Emflaza to improve access to this treatment. We have and always will support you in that endeavor." [1]

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